Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

NCTID	NCT06747273 (View at clinicaltrials.gov)
Description	The primary objective of this study is to evaluate the safety of SRP-9004. (Show More)
Development Status	Inactive
Indication	Limb-Girdle Muscular Dystrophy, Type 2D/R3
Disease Ontology Term	DOID:0110278
Compound Name	SRP-9004
Compound Alias	Patidistrogene bexoparvovec
Compound Description	rAAVrh74-tMCK-SGCA
Sponsor	Sarepta Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	4 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	SGCA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh74
Editor Type	none
Dose 1	Phase 1/2 study: 1E12 vg/kg/single limb (n=1)
Dose 2	Phase 1/2 study: 1E12 vg/kg/limb (n=3), total dose: 2E12 vg/kg
Dose 3	Phase 1/2 study: 3E12 vg/kg/limb (n=2), total dose: 6E12 vg/kg
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Placed on clinical hold due to patient death, Sarepta announced they were pausing this program as part of a strategic restructuring